Study to Evaluate Effects of Emixustat Hydrochloride in Subjects With Proliferative Diabetic Retinopathy

NCT ID: NCT02753400

Last Updated: 2021-05-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-05-31
• Study Completion Date: 2017-12-31

Brief Summary

To evaluate the effects of oral emixustat hydrochloride (emixustat) on aqueous humor biomarkers associated with proliferative diabetic retinopathy (PDR) from baseline to week 12.

Detailed Description

This is a multicenter, randomized, double-masked, placebo-controlled study to evaluate the effects of emixustat in subjects with PDR. Subjects will be randomly assigned to either emixustat or placebo arms and treated once daily (QD) for 12 weeks. Doses of emixustat will be doubled on a weekly basis until week 4 after which all subjects will be held at a stable dose for the remainder of the 12-week dosing regimen. Subjects in the placebo group will be mock-titrated on the same schedule as those in the emixustat arm.

Conditions

Proliferative Diabetic Retinopathy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Emixustat hydrochloride

Week 1- Four tablets (2 placebo, 2 emixustat HCl Strength A)

Week 2- Four tablets (2 placebo, 2 emixustat HCl Strength B)

Week 3- Four tablets (2 placebo, 2 emixustat HCl Strength C)

Week 4- Four emixustat HCl tablets (Strength C)

All tablets are administered orally once daily. After week 4, all subjects will be held at a stable dose for the remainder of the 12-week dosing regimen.

Group Type: EXPERIMENTAL

emixustat hydrochloride

Intervention Type: DRUG

Tablet for oral administration

Placebo

Intervention Type: OTHER

Placebo tablets for oral administration contain only inactive ingredients

Placebo

Four placebo tablets are administered orally once daily for 12 weeks; Subjects in the placebo group will be mock-titrated on the same schedule as those in the active arm.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

Placebo tablets for oral administration contain only inactive ingredients

Interventions

emixustat hydrochloride

Tablet for oral administration

Intervention Type: DRUG

Placebo

Placebo tablets for oral administration contain only inactive ingredients

Intervention Type: OTHER

Other Intervention Names

ACU-4429, Emixustat HCl

Eligibility Criteria

Inclusion Criteria

• Able and willing to provide written informed consent
• Documented diagnosis of type 1 or type 2 diabetes mellitus
• Meets specific ocular criteria for the study eye including but not limited to, the presence of PDR with or without diabetic macular edema in study eye for which treatment can be deferred for at least 4 weeks after Day 1 visit
• Media clarity, pupillary dilation, and subject cooperation sufficient to obtain adequate assessments

Exclusion Criteria

• Any condition that would preclude participation in the study (e.g., unstable medical status including blood pressure, cardiovascular disease or glycemic control)
• History of myocardial infarction or other acute cardiac event
• History of chronic renal failure requiring dialysis or kidney transplant
• Prior participation in any clinical study of emixustat
• Treatment with any investigational study drug within 30 days of screening
• Known allergy to fluorescein sodium for injection in angiography
• Treatment with specific prohibited medications or therapy beginning 4 weeks prior to screening and throughout the duration of the study
• History of systemic anti-VEGF or pro-VEGF treatment within 4 months prior to randomization
• Pre-specified laboratory abnormalities at screening
• Specific ocular characteristics in the study eye
• Male subjects who are not surgically sterile and are not willing to practice a medically accepted method of birth control with their female partner of childbearing potential from screening through 30 days following completion of the study
• Female subjects of childbearing potential who are not willing to practice a medically accepted method of birth control with their non-surgically sterile male sexual partner from screening through 30 days following completion of the study
• Female subjects who are pregnant or lactating

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Kubota Vision Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Responsible Medical Officer

Role: STUDY_DIRECTOR

Kubota Vision Inc.

Locations

Retina Institute of California

Arcadia, California, United States

Countries

United States

References

Kubota R, Jhaveri C, Koester JM, Gregory JK. Effects of emixustat hydrochloride in patients with proliferative diabetic retinopathy: a randomized, placebo-controlled phase 2 study. Graefes Arch Clin Exp Ophthalmol. 2021 Feb;259(2):369-378. doi: 10.1007/s00417-020-04899-y. Epub 2020 Aug 27.

Reference Type: DERIVED

PMID: 32852613 (View on PubMed)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

4429-203

Identifier Type: -

Identifier Source: org_study_id